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US 12,735,723 B2Granted
Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsLouise Rodino-Klapac, Jerry R. Mendell
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Abstract
Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) scAAVrh74.tMCK.hSGCA vector, methods of expressing alpha-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
Claims18
A polynucleotide sequence comprising the nucleotide sequence at least 95% identical to the nucleotide sequence of SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.